tbuckley/cabot-search huggingface dataset:
PB2492: ASSESSMENT OF HEMATOLOGICAL PARAMETERS OF PETROL FILLING WORKERS AT PETROL STATIONS IN GONDAR TOWN, NORTHWEST ETHIOPIA: A COMPARATIVE CROSS-SECTIONAL STUDY
Authors: Sisay Getu, Mulugeta Melku, Elias Shiferaw
Journal: HemaSphere (2023)

Topic: 23. Hematopoiesis, stem cells and microenvironment Background: Petrol is the non-specific term for petroleum which is used for the inside combustion of engines. Petrol filling workers are highly vulnerable to occupational exposure to these harmful substances which lead to hematotoxicity and blood disorders such as leukemia, aplastic anemia, and dysplastic bone marrow. Aims: This study aimed to assess the hematological parameters of petrol-filling workers in Gondar town, Northwest Ethiopia. Methods: A comparative cross-sectional study was conducted from January to March 2019 in Gondar town, Northwest Ethiopia. A total of 110 study participants comprising 55 study groups and 55 controls group were recruited by a convenient sampling technique. Socio-demographic data were collected using a structured questionnaire, and 3 ml of venous blood was collected for the determination of hematological parameters. The data were entered into Epi info 7.2.0.1 and analyzed by SPSS version 20. Mean standard deviation, median, and interquartile ranges were used to present the data. Independent t-test and Mann-Whitney U test were used to compare the mean or median difference between parametric and non-parametric hematological parameters, respectively. Moreover, Pearson product-moment and Spearman’s rank-order bivariable correlations analyses were used to describing the correlation between hematological parameters and duration of exposure to petrol. A P value of ≤ 0.05 was considered statistically significant. Results: The study revealed that the mean red blood cell count and hemoglobin level as well as the median hematocrit, mean cell hemoglobin concentration, platelet count, absolute lymphocytes count, and red cell distribution width values of petrol filling workers showed significant increment compared with the control group. On the other hand, the mean cell hemoglobin value of petrol-filling workers showed a significant decrement compared with healthy controls. Moreover, the duration of exposure to petrol showed a significant positive correlation with red blood cell count and mean cell hemoglobin concentration; however, a significant negative correlation was observed with mean cell volume. Summary/Conclusion: This study showed that the majority of hematological parameters of petrol filling workers showed an increment compared with healthy controls which might be associated with exposure to petrol chemicals. However, further longitudinal studies with a larger sample size should be conducted to explore the impact of petrol exposure on hematopoiesis. Keywords: Bone marrow failure, Absolute lymphocyte count
PB2532: VARIABLE HEMATOLOGICAL AND CLINICAL PHENOTYPES IN DOUBLE HETEROZYGOTES OF ΒETA-THALASSEMIA AND TRIPLICATED ΑLPHA-GLOBIN GENES
Authors: Polyxeni Delaporta, Christina Vrettou, Vasiliki Chouliara, Maria-Ioanna Chatzieleftheriou, Dimitra Kyriakopoulou, Maria Moraki, Joanne Traeger-Synodinos, Antonis Kattamis
Journal: HemaSphere (2023)

Topic: 27. Thalassemias Background: The excess of unpaired alpha-globin chains is a major determinate of hematological and clinical severity of beta-thalassemia. In beta-thalassemia heterozygotes, the coinheritance of additional alpha-globin genes, most commonly through double heterozygosity for a triplicated alpha-globin gene allele, may exacerbate the clinical and hematological phenotype. Aims: We present the clinical and hematological findings in a cohort of cases with co-inheritance of a triplicated alpha-globin gene allele and heterozygous beta-thalassemia. Methods: Following analysis of alpha and beta globin genotypes 43 cases were referred to our thalassemia clinic with mild to moderate anemia for phenotypic evaluation and clinical follow-up. Results: Data are summarized in Table I. All patients had the αααanti-3.7 allele and were heterozygotes for a β-thalassemia mutation. The most common HBB variant was HBB:c.118C>T (CD39 C>T) (32.6%). Mean Hb levels were 10.1±1.3 g/dl (male 10.6±1.2 g/dl; female 9.8± 1.3 g/dl), with a median of 9.95 g/dl (range: 7.1–12.8 g/dl) (male: 10.75 g/dl; female: 10.75 gr/dl). Five cases (11.6%), had less than 9 gr/dl hemoglobin. Nineteen (44%) had splenomegaly and two had undergone splenectomy with improvement of clinical status. Four cases (9%) had cholecystectomy. Other morbidities included hypothyroidism (3 cases), diabetes mellitus (1 case), osteopenia (6 cases) and osteoporosis (2 cases). Five cases had more than 3 transfusions due to pregnancy, splenectomy or infections. Αll transfused cases had severe beta-thalassemia variants. Five cases developed iron overload and received iron chelation therapy, one of whom was additionally homozygous for the p.Cys282Tyr (C282Y) variant of the HFE gene. Of note is that 35/42 cases carry a null HBB variant and overall 40/42 cases had severe HBB variants. Summary/Conclusion: Double heterozygosity for a beta-thalassemia and a triplicated alpha-globin gene underlies a variable clinical phenotype, ranging from thalassemia minor to thalassemia intermedia of moderate severity, potentially modified by other genetic and environmental factors. These findings indicate that all double heterozygotes should be routinely referred for long-term hematological and clinical follow-up even when presenting with thalassemia minor at initial diagnosis.Keywords: Globin gene, Complications, Thalassemia, Phenotype
Ibrutinib plus fludarabine, cyclophosphamide and rituximab (iFCR) as initial treatment in chronic lymphocytic leukemia/ small lymphocytic leukemia with or without TP53 aberrations: a prospective real-world study in Chinese cohort
Authors: Yi Miao, Yeqin Sha, Yi Xia, Shuchao Qin, Rui Jiang, Luomengjia Dai, Hui Shen, Tonglu Qiu, Wei Wu, Jingyan Qiu, Yilian Yang, Chongyang Ding, Yujie Wu, Lei Fan, Wei Xu, Jianyong Li, Huayuan Zhu
Journal: Blood Cancer Journal (2023)

Ibrutinib plus fludarabine, cyclophosphamide and rituximab (iFCR) as initial treatment in chronic lymphocytic leukemia/ small lymphocytic leukemia with or without TP53 aberrations: a prospective real-world study in Chinese cohort
PB2166: PRIMARY MYELOFIBROSIS: SERUM LACTATE DEHYDROGENASE AS A PREDICTOR OF EARLY LEUKEMIC PROGRESSION
Authors: Pedro Moura, Diogo Oliveira, Anabela Ribeiro, José Carda
Journal: HemaSphere (2023)

Topic: 15. Myeloproliferative neoplasms - Biology & Translational Research Background: Primary Myelofibrosis (PMF) is a myeloproliferative neoplasm commonly associated with an unfavorable prognosis, seeing as the most alarming complication relies on PMF ability to progress to Acute Myeloid Leukemia. The latest evidence suggests that increased levels of serum lactate dehydrogenase (LDH), at the time of initial diagnosis of PMF, have a significant correlation with poor prognosis and leukemic progression (LP). However, no exact cut-off value was ever established for serum LDH usage in PMF prognostication Aims: Our study aims to characterize a cohort of patients with PMF, diagnosed in a central hospital between 2010 and 2017, with follow-up to 2022, and investigate a possible correlation between increased serum LDH levels and LP as an early event, i.e., within the first 5 years after initial diagnosis of PMF. Subsequently, we intend to establish the optimal cut-off value for clinical practice. Methods: All data was collected from clinical files, this being a retrospective, observational and single-center study. Patients were selected based on their meeting the World Health Organization criteria for diagnosis of PMF, having a minimum follow-up time of 5 years and availability of serum LDH level at the time of initial diagnosis. In order to study serum LDH ability to predict LP at 5 years, variables that incorporate prognostic scores were also collected. Univariate and multivariate logistic regression statistics, and a Receiver Operating Characteristics (ROC) analysis curve were performed in order to assess the effect of increased serum LDH on the likelihood of LP in 5 years’ time and establish the optimal cut-off point. Results: A total of 53 patients met the above-outlined criteria, having a median age of 68 years, 62.3% males, a median serum LDH level of 414 U/L, with LP within 5 years occurring in 15.1% of patients. In univariate analysis, the likelihood of LP in 5 years’ time increased 0.6% by each increasing U/L of serum LDH [odds ratio (OR) 1.006, p=0.010]. In multivariate analysis, only including Dynamic Prognostic Scoring System (DIPSS) variables, serum LDH retained its significance (OR 1.007, p=0.025). A cut-off value for serum LDH was calculated at 522 U/L, with a sensitivity and specificity of 75% and 80%, respectively. Summary/Conclusion: We demonstrated a positive correlation between increased serum LDH and LP within the first 5 years after initial diagnosis of PMF, subsequently establishing that serum LDH levels ≥ 522 U/L independently predict LP in 5 years’ time, with the highest achievable sensitivity and specificity. Keywords: Myelofibrosis, Acute myeloid leukemia, Prognosis
PB2474: CONSTRUCTION OF NURSING SCHEME FOR PATIENTS UNDERGOING HEMATOPOIETIC STEM CELL TRANSPLANTATION TO PREPARE FOR DISCHARGE
Authors: LI Wang, Xiaoli Chen, Fangfang Wu, Hengrui Sun, Yimei Feng, XI Zhang
Journal: HemaSphere (2023)

Topic: 22. Stem cell transplantation - Clinical Background: Thalassemia is the most common and harmful single-gene genetic disease in southern China. There are about 350 million thalassemia gene carriers in the world, including 30 million in China. At present, there are no specific drugs and mature gene therapy methods for thalassemia. Hematopoietic stem cell transplantation is the only time-proven method for the clinical cure of moderate and severe thalassemia, and the quality of life of children is our key concern. Aims: This study focuses on the process of discharge preparation for patients with hematopoietic stem cell transplantation, and based on the current status and characteristics of discharge guidance, constructs a set of discharge preparation nursing scheme to guide clinical nurses to implement discharge guidance. Methods: 1. Cross-sectional survey of the nursing plan for patients undergoing hematopoietic stem cell transplantation: cluster sampling method was used to investigate the current situation of patients undergoing hematopoietic stem cell transplantation in our department, and the assessment tools such as “Discharge Readiness Scale” and “Discharge Guidance Quality Scale” were used to analyze the related factors affecting discharge readiness. 2. Qualitative research on the nursing plan for patients undergoing hematopoietic stem cell transplantation: 10 patients discharged from our department were selected and 3 nurses conducted qualitative interviews. 3. Construction of the nursing plan for patients undergoing hematopoietic stem cell transplantation: The draft of the nursing plan for patients undergoing hematopoietic stem cell transplantation was preliminarily drawn up. Combined with the results of two rounds of expert meetings, the plan was adjusted to form the final draft. 4, hematopoietic stem cell transplantation patients discharged from hospital to prepare nursing plan of nurse training. To explore the influence of this scheme on the discharge readiness, the incidence of transplantation complications, quality of life and patient satisfaction of hematopoietic stem cell transplantation patients. Results: Hematopoietic stem cell transplantation patients were carried out for 12 months. Before and after the intervention, the patients’ readiness for discharge, the incidence of transplant-related complications, quality of life and patient satisfaction were statistically significant (P < 0.05). Summary/Conclusion: The nursing plan for patients with hematopoietic stem cell transplantation is designed to help them improve their readiness for discharge by improving the quality of discharge guidance, so that patients with hematopoietic stem cell transplantation can master self-observation and self-care skills more comprehensively, improve their coping ability after discharge and improve their quality of life. Keywords: Hematopoietic cell transplantation
P1113: COMPARATIVE EFFICACY OF ZANUBRUTINIB (ZANU) VERSUS RITUXIMAB (RTX) IN RELAPSED MARGINAL ZONE LYMPHOMA (MZL): MATCHING-ADJUSTED INDIRECT COMPARISON (MAIC)
Authors: Catherine Thieblemont, Kaijun Wang, Sam Keeping, Ina Zhang, Keri Yang, Boxiong Tang, Leyla Mohseninejad
Journal: HemaSphere (2023)

Topic: 36. Ethics and health economics Background: ZANU is a Bruton tyrosine kinase inhibitor that has been evaluated for relapsed/refractory MZL in two phase 2, single-arm (MAGNOLIA, n=66, NCT03846427; BGB-3111-AU-003, n=20, NCT02343120). At 28 and 35 months of study follow-up in MAGNOLIA and BGB-3111-AU-003, respectively, median progression-free survival (PFS) and overall survival (OS) were not reached. Aims: Here, we conducted an unanchored MAIC to estimate relative treatment effects of ZANU versus RTX, a commonly used treatment for patients with relapsed MZL. Note the comparison was restricted to the relapsed population because RTX refractory patients would not be retreated with RTX. Methods: The MAIC was performed using pooled individual patient-level data from MAGNOLIA and BGB-3111-AU-003. Study level-data of RTX in relapsed patients were used from CHRONOS-3 (Özcan et al., Ann Oncol 2021) which was identified as the most suitable comparator study via a systematic literature review. A logistic propensity score model was used to estimate weights for patients in the ZANU trials such that their weighted mean baseline characteristics matched those of CHRONOS-3. The following characteristics were identified as key prognostic factors and included in the base case propensity score model: prior lines of therapy, MZL subtype, relapse after prior therapy, and age. Sensitivity analyses incorporating additional characteristics were not possible owing to a lack of reporting from CHRONOS-3, but the impact of each covariate in the base model was explored via a leave-one-out analysis. Comparisons were conducted for OS, PFS, and objective response rate (ORR) by independent review committee using weighted statistical models, with relative treatment effects presented as hazard ratios (HRs), odds ratios (ORs), and 95% confidence intervals (CIs). Results: After applying weights estimated from the base case propensity score model, the effective sample size (ESS) for ZANU was 39. ZANU significantly reduced the risk of progression (HR 0.29; 95% CI 0.13–0.65, P=0.003) and had a higher probability of response (OR 5.09; 95% CI: 1.84–14.08, P=0.0017) when compared with RTX (Table). OS was comparable for ZANU and RTX, which is consistent with the survival expectancy for indolent lymphomas. The leave-one-out analysis showed that removing any of the characteristics from the propensity score model yielded comparable results. Summary/Conclusion: MAIC results suggest ZANU is associated with improved PFS and ORR compared with RTX in relapsed MZL.Keywords: Marginal zone, Tyrosine kinase inhibitor, Indolent non-Hodgkin’s lymphoma, relapsed/refractory
P916: LONG-TERM OUTCOMES WITH ISATUXIMAB-CARFILZOMIB-DEXAMETHASONE (ISA-KD) IN RELAPSED MULTIPLE MYELOMA PATIENTS WITH 1Q21+ STATUS: UPDATED RESULTS FROM THE PHASE 3 IKEMA STUDY
Authors: Thierry Facon, Philippe Moreau, Ivan Špicka, Kenshi Suzuki, Kwee Yong, Joseph Mikhael, Taro Fukao, Kamlesh Bisht, Nicole Armstrong, Sandrine Macé, Marie-Laure Risse, Thomas Martin
Journal: HemaSphere (2023)

Topic: 14. Myeloma and other monoclonal gammopathies - Clinical Background: Gain or amplification of 1q21 (1q21+, ≥3 copies), a chromosomal abnormality frequently observed in multiple myeloma (MM), has a negative impact on prognosis due to its potential involvement in resistance to MM therapy and disease progression. In the prespecified, long-term analysis of the Phase 3 IKEMA trial in relapsed MM patients (pts), treatment with Isa-Kd showed continued, significant improvement in progression-free survival (PFS) vs Kd (HR 0.58; 95.4% CI 0.42–0.79), with meaningful increase in depth of response (complete response or better [≥CR] 44.1% vs 28.5%; minimal residual disease negativity [MRD-] 33.5% vs 15.4%, MRD- ≥CR 26.3% vs 12.2%), and a manageable safety profile. Aims: In this subgroup analysis of IKEMA, we evaluated efficacy of Isa-Kd in pts with 1q21+ status (with or without high- risk chromosomal abnormalities [HRCA]) and related subgroups – isolated 1q21+ (≥3 copies without HRCA), gain(1q21), amp(1q21) – at long-term follow-up (44.2 months) (NCT03275285). Methods: Pts with 1–3 prior lines of therapy were randomized to Isa-Kd (n=179) or Kd (n=123). Assessment was prespecified (at 30% cutoff by FISH) for 1q21+ status as ≥3 copies, gain(1q21) as 3 copies, and amp(1q21) as ≥4 copies. Results: In the Isa-Kd and Kd arms, 41.9% and 42.3% of pts had 1q21+ status, 26.3% and 25.2% isolated 1q21+, 24.0% and 30.1% gain(1q21), 17.9% and 12.2% amp(1q21) respectively. Greater PFS benefit was achieved with Isa-Kd vs Kd in pts with 1q21+ status (HR 0.58, 95% CI 0.37–0.92) and in pts with isolated 1q21+, gain(1q21), or amp(1q21) (Table). Responses deepened by adding Isa to Kd, with increased rates of very good partial response or better (≥VGPR), ≥CR, MRD-, and MRD- ≥CR (Table). TableSummary/Conclusion: 1q21 abnormalities affect PFS in MM pts. Our results at long-term follow- up of pts with 1q21+ status (with or without HRCA) in the IKEMA study continue to show greater PFS benefit and deeper responses with Isa-Kd than Kd, consistent with the overall population and earlier 1q21+ subgroup interim analyses. Thus, they support Isa-Kd as an effective treatment option also for difficult-to-treat, 1q21+ pts with relapsed MM. Keywords: Multiple myeloma, Chromosomal abnormality, Minimal residual disease (MRD)
P1325: EFFICACY AND SAFETY OF HEROMBOPAG FOR THE TREATMENT OF SECONDARY FAILURE OF PLATELET RECOVERY AFTER ALLOGENEIC HEMATOPOIETIC STEM CELL TRANSPLANTATION
Authors: Lu Wang, Lidan Zhu, Peiyan Kong, Yimei Feng, Ting Chen, Jia Liu, Cheng Zhang, Huanfeng Liu, LI Gao, Shichun Gao, Han Yao, Yuqing Liu, Lu Zhao, Lei Gao, XI Zhang
Journal: HemaSphere (2023)

Topic: 22. Stem cell transplantation - Clinical Background: Thrombocytopenia is a frequent and serious complication after Allogeneic hematopoietic stem cell transplantation (allo-HSCT). It often has a multifactorial etiology, including poor graft function, GVHD, drugs, infections, and microangiopathy. Prolonged thrombocytopenia after HSCT, which is an independent adverse prognostic factor for transplant-related mortality (TRM) and overall survival, has not yet been systematically investigated. Among then, secondary failure of platelet recovery (SFPR) is the most common type of thrombocytopenia after allo-HSCT. SFPR refers to thrombocytopenia that develops after initial platelet engraftment and is not due to graft rejection or relapse. It defined as a decline in platelet count of < 20×109/L for 7 consecutive days or requiring transfusion support after achieving a sustained platelet count > 50×109/L without transfusion for 7 consecutive days after HSCT. Aims: To evaluate the efficacy and safety of herombopag for the treatment of SFPR after allo-HSCT. Methods: Here, we conduct a retrospective study to the efficacy and safety of herombopag in 41 patients with SFPR after allo-HSCT. Patients were administered the following treatment regimen: 5 mg/d herombopag; if the PLT count was less than 50×10^9/L for at least 2 weeks, the dose was increased to 7.5 mg/d; if the PLT count was 200-400×109/L, the dose was reduced; and if the PLT count was greater than 400×109/L, herombopag was terminated. Results: Baseline platelet count of patients before treatment is 11 (3-19) ×109/L. The initial time of treatment is 5 (3-11) months after allo-HSCT, and the median duration of treatment is 92 (35-224) days. Among the 41 patients, 31 patients (75.6%) had complete response (CR, defined as PLT≥50×109/L without PLT transfusion for 7 continuous days), 4 patients (9.8%) had a partial response (PR, defined as PLT of [20-50] ×109/L without PLT transfusion for 7 continuous days), and 6 patient (14.6%) had no response (NR, defined as the application of the maximum tolerated dose for 8 weeks and PLT<20×109/L or the need for PLT transfusion). The median time to obtain CR was 46 (15-224) days after treatment. The time to reach 20×109/L<PLT<50×109/L in the 4 patients with PR was 69 (52-276) days after treatment, respectively. One patient died of intracranial hemorrhage and two patients died of severe infection. Summary/Conclusion: Our results indicated that herombopag can promote platelet recovery for patients with SFPR after allo-HSCT, thereby improving the survival rate of patients and improving the quality of life of patients after transplantation, and providing a new method and strategy for the treatment of thrombocytopenia after allo-HSCT. Keywords: HSCT
PB2522: LIPID PROFILE AND CARDIAC FUNCTIONS IN EGYPTIAN SICKLE CELL DISEASE PATIENTS: A SINGLE CENTER STUDY
Authors: Mona El Ghamrawy, Mohamed Samir, Nahla Magdy, Mai Mohamed Abd El Salam
Journal: HemaSphere (2023)

Topic: 26. Sickle cell disease Background: Sickle cell disease (SCD) is a monogenetic disorder resulting from a point mutation in β-globin gene leading to synthesis of abnormal hemoglobin S. SCD patients are at risk of cardiovascular changes including diastolic, and systolic ventricular dysfunction, dilated cardiac chambers, pulmonary hypertension, and elevated peripheral vascular resistance. Alterations in lipid profile have been reported in different SCD patients’ populations. Dyslipidemia may be a potential risk for cardiovascular disease in SCD patients. Aims: To assess the serum lipid profile in a cohort of SCD patients and its possible association with cardiac dysfunction and other disease related complications. Methods: This cross-sectional study included 50 Egyptian steady-state SCD patients (confirmed by hemoglobin electrophoresis) aged >12 years followed up at Pediatric Hematology Outpatient Clinic, Cairo University Children Hospital. Thirty unrelated healthy ethnically- age- and sex- matched subjects were recruited as a control group. All subjects were enrolled after obtaining willingly informed consent from parents. Serum lipid profile including total cholesterol, high-density lipoprotein (HDL), low-density lipoprotein (LDL) and triglyceride (TG) were performed using automated chemistry analyzer Cobas C 311. Cardiac functions were assessed using M- mode and tissue Doppler Echocardiography Results: SCD patients had statistically significant lower HDL, LDL and higher TG levels compared to control group (p<0.001, <0.001 and 0.02 respectively). Forty SCD patients (80%) showed evidence of dyslipidemia. Cardiac assessment showed significantly higher LVlDd, LVlDs, LA diameter, TAPSE, right side Eˋ/cm/s, septal wall E’/cm/s, and left side wall E’/cm/s among SCD compared to control group (p-value <0.001). HDL was significantly lower among SCD patients with cardiac dysfunction (p=0.03). HDL was the only statistically significant predictor of cardiac dysfunction with 74.19% sensitivity, 63.16% specificity, and p-value =0.023. Summary/Conclusion: Serum lipid abnormalities in SCD patients include low HDL, LDL and high TG levels. Low HDL was associated with cardiac dysfunction in our SCD patients. Further prospective multicenter studies are warranted to study the effect of dyslipidemia on cardiovascular changes in SCD. Keywords: Sickle cell disease, Lipid metabolism